WO2021117009A3 - Allele-specific silencing of transforming growth factor beta induced gene with r124h mutation using short interfering rna - Google Patents

Allele-specific silencing of transforming growth factor beta induced gene with r124h mutation using short interfering rna Download PDF

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Publication number
WO2021117009A3
WO2021117009A3 PCT/IB2020/061852 IB2020061852W WO2021117009A3 WO 2021117009 A3 WO2021117009 A3 WO 2021117009A3 IB 2020061852 W IB2020061852 W IB 2020061852W WO 2021117009 A3 WO2021117009 A3 WO 2021117009A3
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Prior art keywords
allele
mutation
growth factor
transforming growth
interfering rna
Prior art date
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PCT/IB2020/061852
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French (fr)
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WO2021117009A2 (en
Inventor
Tara MOORE
Amanda KOWALCZYK
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Moore Tara
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Priority to JP2022536545A priority Critical patent/JP2023505737A/en
Priority to KR1020227023650A priority patent/KR20220113485A/en
Priority to EP20899713.0A priority patent/EP4073095A2/en
Priority to CN202080095670.XA priority patent/CN115515968A/en
Publication of WO2021117009A2 publication Critical patent/WO2021117009A2/en
Publication of WO2021117009A3 publication Critical patent/WO2021117009A3/en

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    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1136—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against growth factors, growth regulators, cytokines, lymphokines or hormones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Endocrinology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Ophthalmology & Optometry (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Abstract

The present disclosure relates to a ribonucleic acid (RNA) complex and methods of use thereof for preventing, ameliorating or treating symptoms associated with granular corneal dystrophy type 2 in a subject.
PCT/IB2020/061852 2019-12-11 2020-12-11 Allele-specific silencing of transforming growth factor beta induced gene with r124h mutation using short interfering rna WO2021117009A2 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
JP2022536545A JP2023505737A (en) 2019-12-11 2020-12-11 Allele-specific silencing of the transforming growth factor beta-inducible gene with the R124H mutation using short interfering RNA
KR1020227023650A KR20220113485A (en) 2019-12-11 2020-12-11 Allele-specific silencing of transforming growth factor beta-induced genes by R124H mutation using short interfering RNA
EP20899713.0A EP4073095A2 (en) 2019-12-11 2020-12-11 Allele-specific silencing of transforming growth factor beta induced gene with r124h mutation using short interfering rna
CN202080095670.XA CN115515968A (en) 2019-12-11 2020-12-11 Allele-specific silencing of transforming growth factor beta-inducible genes with R124H mutation using short interfering RNA

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962946880P 2019-12-11 2019-12-11
US62/946,880 2019-12-11
US202062975713P 2020-02-12 2020-02-12
US62/975,713 2020-02-12

Publications (2)

Publication Number Publication Date
WO2021117009A2 WO2021117009A2 (en) 2021-06-17
WO2021117009A3 true WO2021117009A3 (en) 2021-08-26

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PCT/IB2020/061852 WO2021117009A2 (en) 2019-12-11 2020-12-11 Allele-specific silencing of transforming growth factor beta induced gene with r124h mutation using short interfering rna

Country Status (5)

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EP (1) EP4073095A2 (en)
JP (1) JP2023505737A (en)
KR (1) KR20220113485A (en)
CN (1) CN115515968A (en)
WO (1) WO2021117009A2 (en)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024162652A1 (en) * 2023-02-01 2024-08-08 주식회사 메디치바이오 Pharmaceutical composition using antisense oligonucleotide (aso) for prevention or treatment avellino corneal dystrophy

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060094032A1 (en) * 2004-03-12 2006-05-04 Fougerolles Antonin D iRNA agents targeting VEGF
WO2009152500A2 (en) * 2008-06-13 2009-12-17 University Of Massachusetts Methods and compositions for mediating gene silencing
WO2014175604A2 (en) * 2013-04-23 2014-10-30 주식회사 녹십자엠에스 Composition for diagnosing avellino corneal dystrophy and method for diagnosing avellino corneal dystrophy
US20190040378A1 (en) * 2017-07-04 2019-02-07 Curevac Ag Novel nucleic acid molecules

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060094032A1 (en) * 2004-03-12 2006-05-04 Fougerolles Antonin D iRNA agents targeting VEGF
WO2009152500A2 (en) * 2008-06-13 2009-12-17 University Of Massachusetts Methods and compositions for mediating gene silencing
WO2014175604A2 (en) * 2013-04-23 2014-10-30 주식회사 녹십자엠에스 Composition for diagnosing avellino corneal dystrophy and method for diagnosing avellino corneal dystrophy
US20190040378A1 (en) * 2017-07-04 2019-02-07 Curevac Ag Novel nucleic acid molecules

Also Published As

Publication number Publication date
JP2023505737A (en) 2023-02-10
KR20220113485A (en) 2022-08-12
EP4073095A2 (en) 2022-10-19
WO2021117009A2 (en) 2021-06-17
CN115515968A (en) 2022-12-23

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